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Results for Gene Therapy

1 - 10 of 442

Benjamin J. Samelson-Jones, MD, PhD
Benjamin J. Samelson-Jones

Dr. Samelson-Jones is a pediatric hematologist dedicated to improving the lives of children with bleeding and clotting disorders. His research focuses on gene therapy for hemophilia, the biochemical basis of coagulation, and the immune responses to hemophilia therapies.

E-mail:
samelsonjonesb [at] chop.edu
Lindsey A. George, MD
Lindsey A. George

Dr. George's basic and clinical research interests are in the development of novel therapeutics for hemophilia. Her basic science laboratory studies the molecular basis of coagulation, and she is the principal investigator of ongoing hemophilia A and B gene therapy trials.

E-mail:
georgel [at] chop.edu
Denise E. Sabatino, PhD
Denise E. Sabatino

The research in the Sabatino Laboratory is focused on hemophilia, an inherited bleeding disorder. The interests of the laboratory include the study of variants of coagulation factor VIII to understand the biochemical properties of these proteins and to identify novel variants with enhanced function, and the development of gene-based therapeutic approaches for treating hemophilia.

E-mail:
sabatinod [at] chop.edu
Matthew D. Weitzman, PhD
Matthew D. Weitzman Headshot

Dr. Weitzman's research program aims to understand host responses to virus infection, and the cellular environment encountered and manipulated by viruses. He studies multiple viruses in an integrated experimental approach that combines biochemistry, molecular biology, genetics, and cell biology.

E-mail:
weitzmanm [at] chop.edu
Paris Margaritis, DPhil
Paris Margaritis

Dr. Margaritis uses biochemical, molecular, and complex in vivo methodology within the field of coagulation to advance the understanding of molecular mechanisms involved in pro- and anti-coagulant reactions, and translate research for the treatment of coagulation defects.

E-mail:
margaritis [at] chop.edu
Alan W. Flake, MD
Alan W. Flake

Dr. Flake is a general pediatric surgeon with a clinical and research focus on prenatal treatment ranging from the fetal surgical repair of anatomic anomalies to prenatal stem cell and gene therapy. He has extensive experience in developing rodent, canine, and sheep models for in utero transplantation and for investigating fetal surgery for anatomic malformations.

E-mail:
flake [at] chop.edu
Johannes C.M. van der Loo, PhD
Johannes C.M. van der Loo

Dr. van der Loo is an Adjunct Associate Professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania and Director of the Clinical Vector Core at CHOP, focusing on manufacturing of GMP and GMP process-comparable AAV and Lentiviral vectors for preclinical and gene therapy early phase clinical studies.

E-mail:
vanderlooj [at] chop.edu
Evan Weber, PhD
Evan W. Weber

Dr. Weber is developing approaches to enhance CAR-T cell therapies for pediatric cancer by reprogramming T cells with improved durability and exhaustion resistance. His work will uncover molecular mechanisms that promote CAR-T cell exhaustion and identify new targets for therapeutic intervention.

E-mail:
weberew [at] chop.edu
Stefano Rivella, PhD
Stefano Rivella

Dr. Rivella is an expert in the pathophysiology of erythroid and iron disorders and in the generation of lentiviral vectors for the cure of hemoglobinopathies. He also investigates additional disorders such as anemia of inflammation and hemochromatosis.

E-mail:
rivellas [at] chop.edu
Beverly L. Davidson, PhD
Photo of Beverly L. Davidson, PhD

Dr. Davidson works to understand the molecular basis of childhood onset neurodegenerative diseases and the development of gene and small molecule therapies for treatment. She also focuses on how noncoding RNAs participate in neural development and neurodegenerative disease processes, and how they can be harnessed for therapies.

E-mail:
davidsonbl [at] chop.edu
1 - 10 of 442

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In this section

Filter By

  • Principal Investigator / Faculty (31)
  • Researcher / Lab Personnel (8)
  • Research Administration (1)

Organizations

  • Raymond G. Perelman Center for Cellular and Molecular Therapeutics (57)
  • Department of Pediatrics (46)
  • Division of Oncology (38)
  • Center for Childhood Cancer Research (37)
  • Center for Applied Genomics (34)
  • Division of Hematology (29)
  • Center for Injury Research and Prevention (27)
  • Center for Autism Research (25)
  • Division of Human Genetics (19)
  • PolicyLab (18)
  • Division of Neurology (17)
  • Department of Pathology and Laboratory Medicine (16)
  • Division of Infectious Diseases (15)
  • Department of Biomedical and Health Informatics (13)
  • Center for Computational and Genomic Medicine (12)
  • Center for Mitochondrial and Epigenomic Medicine (12)
  • Cancer Immunotherapy (CAR-T) (11)
  • Division of Allergy and Immunology (11)
  • Cell and Gene Therapy Collaborative (10)
  • Center for Data-Driven Discovery in Biomedicine (10)
  • Mitochondrial Medicine (10)
  • Center for Pediatric Clinical Effectiveness (9)
  • Office of Academic Training and Outreach Programs (8)
  • Children’s Oncology Group (7)
  • Department of Anesthesiology and Critical Care Medicine (7)
  • Department of Child and Adolescent Psychiatry and Behavioral Sciences (7)
  • Rivella Laboratory (7)
  • Roberts Individualized Medical Genetics Center (7)
  • Division of Endocrinology and Diabetes (6)
  • Division of Neonatology (6)
  • Division of Pulmonary and Sleep Medicine (6)
  • Leukodystrophy Center of Excellence (6)
  • Spatial and Functional Genomics Collaborative (6)
  • Anderson Laboratory (5)
  • Clinical In Vivo Gene Therapy (5)
  • Department of Radiology (5)
  • Division of Cardiology (5)
  • Lifespan Brain Institute (5)
  • Maris Laboratory (5)
  • Clinical Vector Core (4)
  • Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders (4)
  • Department of Surgery (4)
  • Division of Adolescent Medicine (4)
  • Division of Emergency Medicine (4)
  • Division of Gastroenterology, Hepatology, and Nutrition (4)
  • Division of Nephrology (4)
  • Division of Neurosurgery (4)
  • Epilepsy NeuroGenetics Initiative (ENGIN) (4)
  • PennCHOP Microbiome Program (4)
  • Bhoj Laboratory (3)
  • Center for Applied Genomics Laboratory (3)
  • Davidson Laboratory (3)
  • Division of Cancer Pathobiology (3)
  • Division of Orthopaedic Surgery (3)
  • Jill and Mark Fishman Center for Lymphatic Disorders (3)
  • Lurie Family Foundations MEG Imaging Center (3)
  • Romberg Laboratory (3)
  • Bernt Laboratory (2)
  • Center for Human Phenomic Science (2)
  • Comprehensive Bone Marrow Failure Center (2)
  • Division of General Pediatrics (2)
  • Division of Neuroradiology (2)
  • eMERGE (2)
  • Intellectual and Developmental Disabilities Research Center (2)
  • Minds Matter Concussion Program (2)
  • Mossé Laboratory (2)
  • PEDSnet (2)
  • Research Vector Core (2)
  • Sleep Center (2)
  • Vaccine and Treatment Evaluation Unit (2)
  • Vanderver Laboratory (2)
  • Wyss/Campbell Center for Thoracic Insufficiency Syndrome (2)
  • Ahrens-Nicklas Laboratory (1)
  • Aquatic Zebrafish Core (1)
  • Bailis Laboratory (1)
  • Beckwith-Wiedemann Syndrome Program of Excellence (1)
  • Bosse Laboratory (1)
  • Brain-Gene-Development Lab (1)
  • Brodeur Laboratory (1)
  • Center for Child Injury Prevention Studies (1)
  • Center for Single Cell Biology (1)
  • Center for the Investigation of Factor FVIII Immunogenicity (1)
  • Center for Violence Prevention (1)
  • Choi Laboratory (1)
  • Chou Laboratory (1)
  • Chronic Kidney Disease in Children Study (1)
  • Congenital Hyperinsulinism Research Laboratory (1)
  • Cystic Fibrosis-Related Diabetes Research Program (1)
  • DBHi Bioinformatics (1)
  • Developmental Biology Research Affinity Group (1)
  • Division of Ophthalmology (1)
  • Division of Plastic and Reconstructive Surgery (1)
  • Division of Protective Immunity (1)
  • Division of Transplant Immunology (1)
  • Division of Urology (1)
  • Fetal Biology and Therapy Research Affinity Group (1)
  • Food Allergy Center (1)
  • Fred and Suzanne Biesecker Pediatric Liver Center (1)
  • Friedreich’s Ataxia Center of Excellence (1)
  • Hamilton Laboratory (1)
  • Hill Laboratory (1)
  • Immune Dysregulation Program (1)
  • Inflammatory Bowel Disease Center (1)
  • Innovation Ecosystem (1)
  • John Laboratory (1)
  • Kurre Laboratory (1)
  • Metabolism, Nutrition, and Physical Development Research Affinity Group (1)
  • Mir Laboratory (1)
  • Mitochondria Research Affinity Group (1)
  • Neuroscience Research Affinity Group (1)
  • Nissim Laboratory (1)
  • Office of Faculty Development (1)
  • Office of Research Safety (1)
  • Office of Technology Transfer (1)
  • Office of the Ruth M. Colket Chair Pediatric Nursing Science (1)
  • Pediatric Center of Excellence in Nephrology (1)
  • Penn-CHOP Kidney Innovation Center (1)
  • Pennsylvania Pediatric Medical Device Consortium (1)
  • Song Laboratory (1)
  • St. Geme Laboratory (1)
  • Stress Neurobiology Program (1)
  • Thomas-Tikhonenko Laboratory (1)
  • Thyroid Center (1)
  • Timko Research Group (1)
  • Translational Research Program in Pediatric Orthopaedics (1)
  • Wolfe Lab (1)

In this section

Filter By

  • Principal Investigator / Faculty (31)
  • Researcher / Lab Personnel (8)
  • Research Administration (1)

Organizations

  • Raymond G. Perelman Center for Cellular and Molecular Therapeutics (57)
  • Department of Pediatrics (46)
  • Division of Oncology (38)
  • Center for Childhood Cancer Research (37)
  • Center for Applied Genomics (34)
  • Division of Hematology (29)
  • Center for Injury Research and Prevention (27)
  • Center for Autism Research (25)
  • Division of Human Genetics (19)
  • PolicyLab (18)
  • Division of Neurology (17)
  • Department of Pathology and Laboratory Medicine (16)
  • Division of Infectious Diseases (15)
  • Department of Biomedical and Health Informatics (13)
  • Center for Computational and Genomic Medicine (12)
  • Center for Mitochondrial and Epigenomic Medicine (12)
  • Cancer Immunotherapy (CAR-T) (11)
  • Division of Allergy and Immunology (11)
  • Cell and Gene Therapy Collaborative (10)
  • Center for Data-Driven Discovery in Biomedicine (10)
  • Mitochondrial Medicine (10)
  • Center for Pediatric Clinical Effectiveness (9)
  • Office of Academic Training and Outreach Programs (8)
  • Children’s Oncology Group (7)
  • Department of Anesthesiology and Critical Care Medicine (7)
  • Department of Child and Adolescent Psychiatry and Behavioral Sciences (7)
  • Rivella Laboratory (7)
  • Roberts Individualized Medical Genetics Center (7)
  • Division of Endocrinology and Diabetes (6)
  • Division of Neonatology (6)
  • Division of Pulmonary and Sleep Medicine (6)
  • Leukodystrophy Center of Excellence (6)
  • Spatial and Functional Genomics Collaborative (6)
  • Anderson Laboratory (5)
  • Clinical In Vivo Gene Therapy (5)
  • Department of Radiology (5)
  • Division of Cardiology (5)
  • Lifespan Brain Institute (5)
  • Maris Laboratory (5)
  • Clinical Vector Core (4)
  • Comprehensive Center for the Cure of Sickle Cell Disease and Other Red Blood Cell Disorders (4)
  • Department of Surgery (4)
  • Division of Adolescent Medicine (4)
  • Division of Emergency Medicine (4)
  • Division of Gastroenterology, Hepatology, and Nutrition (4)
  • Division of Nephrology (4)
  • Division of Neurosurgery (4)
  • Epilepsy NeuroGenetics Initiative (ENGIN) (4)
  • PennCHOP Microbiome Program (4)
  • Bhoj Laboratory (3)
  • Center for Applied Genomics Laboratory (3)
  • Davidson Laboratory (3)
  • Division of Cancer Pathobiology (3)
  • Division of Orthopaedic Surgery (3)
  • Jill and Mark Fishman Center for Lymphatic Disorders (3)
  • Lurie Family Foundations MEG Imaging Center (3)
  • Romberg Laboratory (3)
  • Bernt Laboratory (2)
  • Center for Human Phenomic Science (2)
  • Comprehensive Bone Marrow Failure Center (2)
  • Division of General Pediatrics (2)
  • Division of Neuroradiology (2)
  • eMERGE (2)
  • Intellectual and Developmental Disabilities Research Center (2)
  • Minds Matter Concussion Program (2)
  • Mossé Laboratory (2)
  • PEDSnet (2)
  • Research Vector Core (2)
  • Sleep Center (2)
  • Vaccine and Treatment Evaluation Unit (2)
  • Vanderver Laboratory (2)
  • Wyss/Campbell Center for Thoracic Insufficiency Syndrome (2)
  • Ahrens-Nicklas Laboratory (1)
  • Aquatic Zebrafish Core (1)
  • Bailis Laboratory (1)
  • Beckwith-Wiedemann Syndrome Program of Excellence (1)
  • Bosse Laboratory (1)
  • Brain-Gene-Development Lab (1)
  • Brodeur Laboratory (1)
  • Center for Child Injury Prevention Studies (1)
  • Center for Single Cell Biology (1)
  • Center for the Investigation of Factor FVIII Immunogenicity (1)
  • Center for Violence Prevention (1)
  • Choi Laboratory (1)
  • Chou Laboratory (1)
  • Chronic Kidney Disease in Children Study (1)
  • Congenital Hyperinsulinism Research Laboratory (1)
  • Cystic Fibrosis-Related Diabetes Research Program (1)
  • DBHi Bioinformatics (1)
  • Developmental Biology Research Affinity Group (1)
  • Division of Ophthalmology (1)
  • Division of Plastic and Reconstructive Surgery (1)
  • Division of Protective Immunity (1)
  • Division of Transplant Immunology (1)
  • Division of Urology (1)
  • Fetal Biology and Therapy Research Affinity Group (1)
  • Food Allergy Center (1)
  • Fred and Suzanne Biesecker Pediatric Liver Center (1)
  • Friedreich’s Ataxia Center of Excellence (1)
  • Hamilton Laboratory (1)
  • Hill Laboratory (1)
  • Immune Dysregulation Program (1)
  • Inflammatory Bowel Disease Center (1)
  • Innovation Ecosystem (1)
  • John Laboratory (1)
  • Kurre Laboratory (1)
  • Metabolism, Nutrition, and Physical Development Research Affinity Group (1)
  • Mir Laboratory (1)
  • Mitochondria Research Affinity Group (1)
  • Neuroscience Research Affinity Group (1)
  • Nissim Laboratory (1)
  • Office of Faculty Development (1)
  • Office of Research Safety (1)
  • Office of Technology Transfer (1)
  • Office of the Ruth M. Colket Chair Pediatric Nursing Science (1)
  • Pediatric Center of Excellence in Nephrology (1)
  • Penn-CHOP Kidney Innovation Center (1)
  • Pennsylvania Pediatric Medical Device Consortium (1)
  • Song Laboratory (1)
  • St. Geme Laboratory (1)
  • Stress Neurobiology Program (1)
  • Thomas-Tikhonenko Laboratory (1)
  • Thyroid Center (1)
  • Timko Research Group (1)
  • Translational Research Program in Pediatric Orthopaedics (1)
  • Wolfe Lab (1)
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